Cystic FibrosisLung Transplantation | Asthma | Lung Cancer | PCD | Critical Care | Airway Biology | COPD/Chronic Bronchitis/Emphysema | Pulmonary Infections | Pulmonary HypertensionSarcoidosis | Bronchiectasis | Interstitial Lung Disease

Bronchiectasis

Idiopathic Bronchiectasis and Dural Ectasia: Is there a correlation? IRB Study #11-1997

Sponsor: UNC NCTRACS

PI: Leigh Anne Daniels, MD
Co-Investigators: Mike Knowles, MD

To determine if there is a connection between idiopathic bronchiectasis and connective tissue disorders, this study will look to see if dural ectasia is more common in patients with idiopathic bronchiectasis, as compared to patients of bronchiectasis of known causes and patients without bronchiectasis.

Contact: Leigh Anne Daniels, MD

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multi-Center Study to Assess the Efficacy, Safety and Tolerability, and Pharmacokinetics of INS1007 Administered Once Daily for 24 Weeks in Subjects with Non-Cystic Fibrosis Bronchiectasis – The Willow Study

 Sponsor: Insmed Incorporated
Principal Investigator: Peadar Noone, MD

Phase 2 randomized, double-blind, placebo-controlled, parallel-group, multicenter, multi-national study to assess the efficacy, safety and tolerability, and pharmacokinetics (PK) of INS1007 administered once daily for 24 weeks in subjects with non-cystic fibrosis bronchiectasis (NCFBE).

Contact: Samantha Earnhardt, MPH CCRC  & Sonya Capps

Long Term Efficacy and Safety of Inhaled Colistimethate Sodium in Bronchiectasis Subjects With Chronic Pseudomonas Aeruginosa Infection

Sponsor: Zambon SpA
Principal Investigator: Leigh Anne Daniels, MD MPH

The co-primary objectives of the trial are to investigate the effect of the use of inhaled colistimethate sodium, administered twice daily via the I-neb for 12 months, compared to placebo in subjects with non-cystic fibrosis bronchiectasis (NCFB) chronically infected with P. aeruginosa on the frequency of pulmonary exacerbations; the number of exacerbation-free days.

Contact : Samantha Earnhardt, MPH CCRC  & Sonya Capps

Multi-center randomized pragmatic clinical trial comparing 2- versus 3-antibiotic therapy for pulmonary Mycobacterium avium complex disease

Sponsor: PCORI
Principal Investigator: Peadar Noone, MD

This multi-center pragmatic randomized clinical trial will compare 2- versus 3-drug therapy for patients with pulmonary Mycobacterium avium complex (MAC) infection to better understand the real-world effectiveness and tolerability of these treatments. Patients will be randomized to azithromycin + ethambutol (2-drug therapy) vs. azithromycin + ethambutol + rifampin (3-drug therapy). Patients with cavitary disease may additionally receive 2 months of intravenous amikacin

Contact : Samantha Earnhardt, MPH CCRC  & Sonya Capps

COPD/Chronic Bronchitis/Emphysema

Cathelicidin and Vitamin D: Impact on Populations with COPD – IRB Study #17-2170

Sponsor: National Institutes of Health

PI: Brad Drummond, M.D.
Co-Investigators: Ashley Henderson, M.D.

The purpose of this study is to determine the effect of vitamin D (given by mouth) on a marker of inflammation measured in the lungs and blood. Eligible participants include current or former smokers with COPD and low vitamin D level. Interested individuals will complete a screening questionnaire, undergo breathing tests (if not available in the last year), and have blood tests to measure vitamin D levels. If they qualify, they will then have blood collected and undergo a bronchoscopy (lighted camera into lungs under twilight sedation) to collect inflammation levels before and after 8 weeks of oral vitamin D treatment.

Contact: 

UNC Smoking-Related Lung Diseases Registry and Biorepository
IRB Study #16-3187

Sponsor: UNC Division of Pulmonary Medicine

PI: Brad Drummond, M.D.
Co-Investigators: Ashley Henderson, M.D.

The purpose of this study is to provide a registry of smokers and those with COPD who are interested in participating in future clinical trials. Participants will answer a brief questionnaire, undergo lung function tests (if not recently completed), and give a small amount of blood for storage. The information will be used to identify appropriate studies for interested patients who smoke or who have COPD. The stored blood samples will provide a resource for testing of mechanisms of lung disease development.

Contact:

Cathelicidin and Vitamin D: Impact on Populations at Risk for COPD
IRB Study #16-2200

Sponsor: National Institutes of Health

PI: Brad Drummond, M.D.
Co-Investigators: Ashley Henderson, M.D.

The purpose of this study is to determine the effect of vitamin D (given by mouth) on a marker of inflammation measured in the lungs and blood. Eligible participants include smokers with normal lung function and low vitamin D level. Interested individuals will complete a screening questionnaire, undergo breathing tests, and have blood tests to measure vitamin D levels. If they qualify, they will then have blood collected and undergo a bronchoscopy (lighted camera into lungs under twilight sedation) to collect inflammation levels before and after 8 weeks of oral vitamin D treatment.

Contact:

ACT15104- A randomized, double-blind, placebo-controlled, parallel-group, Proof-of-Concept (PoC) study to assess the efficacy, safety and tolerability of SAR440340, in patients with moderate-to-severe chronic obstructive pulmonary disease (COPD)

Sponsor: Sanofi, Inc
Principal Investigator: M. Brad Drummond, MD
Co-Investigator: Ashley Henderson, MD

This is a study of a new medication (administered as injection under the skin) to treat patients with COPD with continued flares of their breathing. The study requires visits every two weeks for one year.

Contacts: Karen Hardy, MS CRC and Andrea McDaniel-Harper, LPN CCRC

EFC15804-A Randomized, Double-blind, Placebo-controlled,Parallel-group, 52-week Pivotal Study to Assess the Efficacy, Safety, and Tolerability of Dupilumab in Patients With Moderate-to-severe Chronic Obstructive Pulmonary Disease (COPD) with Type 2 inflammation

Sponsor: Sanofi, Inc
Principal Investigator: M. Brad Drummond, MD
Co-Investigator: Ashley Henderson, MD

This is a study of a medication administered as injection under the skin to treat patients with COPD who have evidence of elevated allergic cells in their blood as well as continued flares of their breathing. The study requires visits every two weeks for one year.

Contacts: Karen Hardy, MS CRC and Andrea McDaniel-Harper, LPN CCRC

A single center, open label, prospective study measuring proportion of patients with suboptimal Peak Inspiratory Flow Rate (sPIFR) over 24 weeks in an ambulatory setting among moderate to very severe COPD patients

Sponsor: Boehringer-Ingelheim
Principal Investigator: M. Brad Drummond, MD

This study seeks to better understand peak inspiratory flow rate in COPD patients receiving dry powder inhaler(s) in the ambulatory setting. Participants will perform home measurements of inspiratory effort three times a week for 6 months, with two in-person clinic visits.

Contacts: Karen Hardy, MS CRC and Andrea McDaniel-Harper, LPN CCRC

 RofLumilast or Azithromycin to preveNt COPD Exacerbations (RELIANCE) Study

 Sponsor: Patient-Centered Outcomes Research Institute (PCORI)
Principal Investigator: M. Brad Drummond, MD

This study is a real-world trial comparing azithromycin to roflumilast in patients recently hospitalized for a flare of COPD. There are no study visits required after enrollment.

Contacts: Karen Hardy, MS CRC and Andrea McDaniel-Harper, LPN CCRC

Cystic Fibrosis

Gene Modifiers in Cystic Fibrosis IRB Study #00-1420

Sponsor: NIH
PI: Mike Knowles, MD

This study has been designed to compare the overall genetic make up of CF patients, in regard to gene modifiers, who are considered to have mild disease versus more severe disease. Blood samples from volunteers will be studied, along with pulmonary function tests and other medical information, in hopes that a connection can be made between genetic make-up and disease severity of CF lung disease.

Contact: cfmod@med.unc.edu

Genetic Modifiers of Inherited Liver Disease IRB Study #01-1421

Sponsor: CFF
PI: Mike Knowles, MD

This study has been designed to compare the overall genetic make up of CF patients, in regard to gene modifiers, who are considered to have mild disease versus more severe disease. Blood samples from volunteers will be studied, along with pulmonary function tests and other medical information, in hopes that a connection can be made between genetic make-up and development of CF liver disease.

Contact: cfmod@med.unc.edu

INVESTIGATOR INITIATED STUDIES:

Xenon vs F19-MRI
Sponsor: NIH
PI: Dr. Jennifer Goralski

This study is designed to compare the capabilities of two novel imaging techniques: polarized perfluorinated gas mixed with oxygen, and hyperpolarized xenon mixed with N2 to detect changes in lung ventilation using MRI.
Contact Info: margret_powell@med.unc.edu
Enrollment: Closed

F19-MRI Study of CF vs. Healthy Lungs
This study is designed to develop a technique to allow visualization of ventilation (ventilation mapping) in the lung using inhaled perfluorpropane as an inhaled MRI contrast agent. Healthy and CF subjects will be enrolled to begin to describe CF specific changes
Sponsor: NIH
PI: Dr. Jennifer Goralski

This study is designed to develop a technique to allow visualization of ventilation (ventilation mapping) in the lung using inhaled perfluorpropane as an inhaled MRI contrast agent. Healthy and CF subjects will be enrolled to begin to describe CF specific changes
Contact Info: margret_powell@med.unc.edu 

F19-MRI Study: Effect of CF Exacerbation Treatment
Sponsor: NIH
PI: Dr. Jennifer Goralski

This study will use F19-MRI to visualize ventilation in CF patients experiencing a pulmonary exacerbation, and again following treatment for that exacerbation.  Correlation to traditional markers of lung disease (e.g. spirometry) will be made.
Contact Info: margret_powell@med.unc.edu

MULTICENTER CLINICAL TRIALS:

Treatment of Pulmonary Exacerbations in people with CF (STOP 2)
Sponsor
: CFF
PI: Dr. Jennifer Goralski

This study is taking place at multiple care centers across the U.S. It will look at the safety and effectiveness of different lengths of IV treatment for pulmonary exacerbations in people with CF.
Contact Info: rcunnion@email.unc.edu

A Phase 1 Study to Evaluate the Saftey, Tolerability, and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults with Cystic Fibrosis
Sponsor: Proteostasis Therapeutics, Inc.
PI: Dr. Scott Donaldson

This study is taking place at multiple care centers across the U.S. It will evaluate the safety, tolerability, and pharmacokinetics of PTI-808 in Healthy Volunteers and Adults in with Cystic Fibrosis.
Contact Info: jd_harris@med.unc.edu

VX17-445-105: A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation 
Sponsor: Vertex Pharmaceuticals Incorporated
PI: Dr. Scott Donaldson

This study is taking place at multiple care centers across the U.S. It will evaluate the long-term safety and tolerability of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with Cystic Fibrosis (CF) who are homozygous or heterozygous for the F508del mutation.
Contact Info: katie_howe@med.unc.edu; alexandria_nesbit@med.unc.edu;  rcunnion@email.unc.edu
Enrollment: Closed   

TEACH: Testing the effect of adding oral azithromycin to inhaled tobramycin in people with CF (TEACH-IP-15)
Sponsor: CFF
PI: Dr. George Retsch-Bogart

This study is taking place at multiple care centers across the U.S. It is for people with CF who are chronically infected with P. aeruginosa and are taking inhaled tobramycin.
Contact Info: rcunnion@email.unc.edu; jd_harris@med.unc.edu

Early MRSA Therapy in CF (Streamlined Eradication with Repeat Cycle) (Staph Aureus Resistance – Treat Early and Repeat (STAR-ter))
Sponsor: CFF
PI: Dr. Marianne Muhlebach

This is an open-label clinical trial being conducted at numerous CF care centers across the US. It will investigate the microbiological efficacy and safety of a streamlined two-week MRSA eradication treatment protocol.
Contact Info: rcunnion@email.unc.edu

To search for additional Clinical Trials that you may be eligible for please go to the Cystic Fibrosis Foundation’s Clinical Trials Finder.

Lung Transplantation

No current studies at this time.

Asthma

A Multicenter, Open-label, Phase 3b Efficacy and Safety Study of Benralizumab 30 mg Administered Subcutaneously to Reduce Oral Corticosteroid Use in Adult Patients with Severe Eosinophilic Asthma on High-Dose Inhaled Corticosteroid plus Long-acting β2 Agonist and Chronic Oral Corticosteroid Therapy (PONENTE)

Sponsor: Astra Zeneca
Principal Investigator: Stephen Tilley, MD

This is an open-label, multicenter study designed to evaluate efficacy and safety of reducing daily oral corticosteroid (OCS) use after initiation of 30 mg dose of benralizumab administered subcutaneously (SC) in patients with severe eosinophilic asthma receiving high-dose inhaled corticosteroid (ICS)/long-acting β2 agonist (LABA) and OCS with or without additional asthma controller(s).

Contact: Corey Jania

Ongoing studies can also been found in the UNC Center for Environmental Medicine, Asthma and Lung Biology.

Lung Cancer

No current studies at this time.

PCD

Research Genetic Testing for Primary Ciliary Dyskinesia Using a Panel of Genes (5905), IRB Study #14-1225

Sponsor: NIH
PI: Mike Knowles, MD

Primary ciliary dyskinesia (PCD) is a Mendelian recessive, genetically heterogeneous disorder with defective mucociliary clearance (MCC), chronic oto-sino-pulmonary disease with bronchiectasis, and organ laterality defects in ~50% of patients (Kartagener Syndrome). Despite the need for early diagnosis and expert clinical care in PCD, establishing a diagnosis remains a major challenge, based on the traditional approaches of using electron microscopy and/or ciliary waveform analysis to define abnormalities of ciliary ultrastructure and/or function. The goal for this study is to determine whether a multi-gene (n=30) genetic test panel to confirm a diagnosis of PCD will identify as many as 70% of PCD patients. If this genetic test panel is successful, it will revolutionize the diagnostic approach in PCD, and lead to early identification and initiation of clinical monitoring and treatment.

Contact:

Longitudinal Study of Primary Ciliary Dyskinesia: Participants 5-18 Years of Age (5901), IRB Study # 05-2997

Sponsor: NIH
PI: Margaret Leigh, MD

Mucociliary clearance (MCC) is the primary defense mechanism for the lung. Inhaled particles (including microbial pathogens) are entrapped in mucus on the airway surface then cleared by the coordinated action of cilia. The volume and composition of airway surface liquid (ASL) influence the efficiency of ciliary function and MCC. Genetic diseases of MCC include disorders in ciliary function (primary ciliary dyskinesia, PCD), and ion transport (cystic fibrosis). The purpose of this longitudinal study protocol is to define age-related prevalence of phenotypic characteristics and the progression of key features of lung disease in subjects with Primary Ciliary Dyskinesia (PCD).

Contact:

Rare Genetic Disorders of the Airways: Cross-sectional Comparison of Clinical Features, and Development of Novel Screening and Genetic Tests (5902), IRB Study # 05-2979

Sponsor: NIH
PI: Mike Knowles, MD

Mucociliary clearance, in which mucus secretions are cleared from the breathing airways, is the primary defense mechanism for the lungs. Inhaled particles, including microbes that can cause infections, are normally entrapped in mucus on the airway surfaces and then cleared out by the coordinated action of tiny hair-like structures called cilia. Individuals with primary ciliary dyskinesia, variant cystic fibrosis, and pseudohypoaldosteronism have defective mucociliary clearance. The purpose of this study is to collect clinical and genetic information about these three airway diseases to improve current diagnostic procedures.

Contact:

CTRC-2589 Early Onset and Progression of Primary Ciliary Dyskinesia Lung Disease Prior to 10 Years of Age (5903), IRB Study # 08-0764

Sponsor: NIH
PI: Margaret Leigh, MD

Primary ciliary dyskinesia (PCD), also known as Kartagener syndrome, is a genetic disorder of the cilia, which are microscopic hair-like cells. Cilia work to keep the respiratory system clean by moving mucus that contains debris to the large airways, where it can be coughed out. People with PCD have cilia that do not move properly and therefore are not effective in cleaning the respiratory system. This study will determine when PCD starts and how it changes over time, specifically in terms of how well the lungs work, what germs grow in lung secretions, and how the lungs look on computed tomography (CT) scans.

Contact: Beth Godwin

Cross-Sectional Characterization of Idiopathic Bronchiectasis (5904), IRB Study #10-1523

Sponsor: NIH
PI: Mike Knowles, MD

Bronchiectasis is a type of lung condition in which the lungs’ airways are abnormally stretched and widened. This stretching and widening makes it difficult for mucus and other substances to move out of the lungs, encouraging the growth of bacteria and leading to breathing problems or infection. Bronchiectasis can be caused by genetic disorders or diseases such as tuberculosis or rheumatoid arthritis. Researchers are interested in developing better ways to diagnose and treat a lung problem called idiopathic or unexplained bronchiectasis. The goal of this study is to better describe the physical characteristics, radiographic patterns, and airway microbiology of unexplained bronchiectasis and to look for possible genetic links or risk factors.

Contact:

Critical Care

Statins for Acutely Injured Lungs from Sepsis (SAILS)

Sponsor: National Institutes of Health, ARDS Network
Site Investigators: Shannon Carson, MD & Lydia Chang, MD

The SAILS study is a multicenter, double-blind, randomized, placebo-controlled clinical trial of rosuvastatin versus placebo comparator for the treatment of patients with ALI or ARDS from suspected sepsis. The hypothesis of this study is that rosuvastatin therapy will improve mortality in patients with sepsis-induced ALI.
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Modifying the Incidence of Neurologic Dysfunction (MIND-USA)

Sponsor: National Institutes of Health
Site Investigators: Shannon Carson, MD & Lydia Chang, MD

The MIND-USA study is a multi-center, double-blind, randomized placebo-controlled trial investigating the effects of haldoperidol and ziprasidone on delirium in at-risk critically ill patients. The hypothesis is that the administration of these commonly used medications will improve short- and long-term clinical outcomes, including days alive without acute brain dysfunction.
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Identifying Effective Strategies to Disclose Prognosis: Physician-Family Communication in Patients with Acute Lung Injury (EC-ALI)

Sponsor: National Institutes of Health
Site Investigators: Shannon Carson, MD & Lydia Chang, MD

The EC-ALI Study is a multi-center, prospective cohort study using both quantitative and qualitative methods to identify effective, acceptable strategies to communicate information about patient care and outcomes to surrogate decision makers. The research plans to determine which communication strategies help accurate understanding and which generate misconceptions about clinical care and outcomes for patients who are critically ill.
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Mortality Prediction Model for Prolonged Mechanical Ventilation (PRO-VENT)

Sponsor: UNC
Principal Investigator: Shannon Carson, MD

Patients requiring prolonged mechanical ventilation (PMV) often survive the initial severe stages of their illness, but remain dependent on life support systems. Long-term prognosis is often confusing or uncertain for patients, families, and physicians. In order to help clarify prognosis for these complicated patients, a prognostic model that identifies PMV patients who have a high risk of mortality was developed and validated in a multi-center study. Currently, the study continues to identify variables for this model to predict prognosis earlier on in a patient’s stay, as well as predict functional outcomes using both qualitative and quantitative methodology.
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Informing Decisions in Chronic Critical Illness (The SIT Study)

Sponsor: National Institutes of Health
Principal Investigator: Shannon Carson, MD

This study is a multi-center, blinded, randomized controlled trial of protocol-driven meetings in which a Supportive Information Team, made up of an interdisciplinary group of palliative care clinicians, provide informational support to families of chronically critically ill patients in order to facilitate communication and decision-making with the ICU physician. The purpose is to learn more about how best to provide support and information to families and evaluate the impact on families of patients on prolonged mechanical ventilation.
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Decision Aid for Improving Decision-making for Patients with Prolonged Mechanical Ventilation. (DST)

Sponsor: National Institutes of Health
Site Investigator: Shannon Carson, MD

This study is a multi-center, randomized, controlled trial (RCT) with 6-month follow up to determine how useful the decision aid would be in improving the quality of the decision making process for patients who are critically ill, their families, and the ICU physicians and nurses. The study plans to measure the effect of the decision aid on physician-family concordance, quality of communication and medical comprehension. The study also aims to measure the decision aid’s effect on post-traumatic stress among family members, and patients’ health care utilization over 6 months.

Airway Biology

Ongoing studies are conducted in the UNC Center for Environmental Medicine, Asthma and Lung Biology.

Pulmonary Infections

BRAVE: Burden of Respiratory Viruses in HIV Infection
Sponsor: National Institutes of Health, Center for AIDS Research
Principal Investigator: William Fischer, MD

 Most infectious respiratory diseases in HIV-infected patients have been attributed to bacterial and fungal infections but little is known about the role respiratory viruses play in HIV infected patients. This study aims to collect excess respiratory samples and clinical information from HIV infected inpatients and outpatients with respiratory symptoms to better understand which viruses affect the lung health of HIV patients, whether HIV disease is a risk factor for severe outcomes in viral illness, and identify novel respiratory viral pathogens missed by current diagnostic standards.

HIRE: The Effect of HIV on the Immune Response to Viral Infection in Human Nasal Epithelial Cells
Principal Investigator: William Fischer, MD

 Emerging evidence suggests that viral pathogens play a significant role in the lung health of HIV infected patients who have both increased susceptibility to and severity of disease despite treatment with anti-retroviral therapy. The goal of this study is to understand how HIV infection affects mucosal inflammation at the level of respiratory epithelial cells, which are the primary target of respiratory viruses such as influenza.

International work

EVD- 001 Evaluation of convalescent plasma in the treatment of Ebola virus disease in Liberia.
Sponsor: Clinical RM
Funding: Bill and Melinda Gates Foundation

This was the first clinical trial of a novel therapeutic agent in an Ebola outbreak.  This trial has ended as there is no active transmission of Ebola virus in West Africa currently.

EVD-002 Collection of Ebola Virus Disease Convalescent Plasma and Longitudinal Clinical and Serosurvey of EVD Survivors
Sponsor: Clinical RM
Funding: Bill and Melinda Gates Foundation

The goal of this project is to establish a longitudinal observational cohort of EVD survivors to prequalify potential ECP donors for ECP donation, collect ECP by apheresis for clinical trials, compassionate use, immune globulin production or other EVD research, and better understand the clinical, viral, and immunologic sequelae of Ebola virus disease.

PREVAIL IV – Double-blind, Randomized, Two-phase, Placebo-controlled, Phase II Trial of GS-5734 to Assess the Antiviral Activity, Longer-term Clearance of Ebola virus, and Safety in Male Ebola Survivors with Evidence of Ebola virus persistence in semen.
Sponsor: National Institutes of Health
Principal Investigators:  William Fischer, Elizabeth Higgs, and Dehkontee Dennis

Following the end of widespread active transmission at least 12 clusters of infections emerged due to the persistence of Ebola virus in survivors of Ebola virus disease. This trial evaluates the efficacy of a novel compound, GS-5734, in reducing shedding of Ebola virus in the semen of EVD survivors.

Pulmonary Hypertension

A Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Safety and Efficacy of Inhaled Treprostinil in Subjects with Pulmonary Hypertension due to Parenchymal Lung Disease

Sponsor: United Therapeutics Corporation
Principal Investigator: H. James Ford III, MD

The primary of objective of the study is  To evaluate the safety and efficacy of inhaled treprostinil in subjects with PH associated with ILD including CPFE.

Contact:   &

An Open-Label Extension study of Inhaled Treprostinil in Subjects with Pulmonary Hypertension due to Parenchymal Lung Disease

Sponsor: United Therapeutics Corporation
Principal Investigator: H. James Ford III, MD

The primary of objective of the study is to evaluate the safety and efficacy of inhaled treprostinil in subjects with PH associated with ILD including CPFE.

Contact: &

BEAT OLE: An Open-label Extension of BPS-314d-MR-PAH-302 in Pulmonary Arterial Hypertension Patients

Sponsor: Lung Biotechnology
Principal Investigator: H. James Ford III, MD

This is a multi-center, open-label study for eligible subjects who were actively participating in the BPS-314d-MR-PAH-302 study at the time the study was concluded. This OLE study will evaluate the safety, tolerability and efficacy of long-term treatment of the esuberaprost sodium tablets (Beraprost Sodium 314d Modified Release tablets) study drug.

Contact: Samantha Earnhardt, MPH CCRC  & Paula Glover, BA

A Phase 3, Open-label, Multicenter Study to Evaluate the Long-term Safety and Tolerability of Inhaled LIQ861 (Treprostinil) in Pulmonary Arterial Hypertension (WHO Group 1) Patients

Sponsor: Liquidia Technologies
Principal Investigator: H. James Ford III, MD

The primary objective of this study is to evaluate the long-term safety and tolerability of LIQ861, a dry powder formulation of treprostinil, in patients with Pulmonary Arterial Hypertension (PAH). A secondary objective of this study is to evaluate the comparative bioavailability of treprostinil between two formulations of inhaled therapy.

Contact: Samantha Earnhardt, MPH CCRC  & Paula Glover, BA

OPUS: US-based, observational, drug registry of Opsumit® (macitentan) new users in clinical practice

Sponsor: Actelion Pharmaceuticals
Principal Investigator: H. James Ford III, MD

This study is a prospective observational drug registry developed to characterize the safety profile (including primarily potential serious hepatic risks) and to describe clinical characteristics and outcomes of patients newly treated with Opsumit in the post-marketing setting.

Contact: Samantha Earnhardt, MPH CCRC  & Paula Glover, BA

Pulmonary Hypertension Association Registry

Sponsor: Pulmonary Hypertension Association
Principal Investigator: H. James Ford III, MD

The goals of the PHAR include 1) measuring and improving quality of care (including assessing differences in adherence to evidence-based guidelines and establishing benchmarks for health outcomes), 2) determining the clinical effectiveness, comparative effectiveness, and cost effectiveness of treatment approaches, 3) understanding risk factors for outcomes and regional/center differences, and 4) facilitating funded clinical trials of new therapies and collaboration with the PAH community at large, including providers, patients, and their caregivers.

Contact: Samantha Earnhardt, MPH CCRC & Paula Glover, BA

Uptravi® (SelexiPag): tHe usErs dRug rEgistry

Sponsor: Actelion Pharmaceuticals
Principal Investigator: H. James Ford III, MD

This is a US multi-center, prospective, real world, observational drug registry enrolling patients actively treated with Uptravi. Participating patients will be followed prospectively for a maximum of 18 months from the date of enrollment into the registry.

Contact: Samantha Earnhardt, MPH CCRC  & Paula Glover, BA

PERFECT: A Phase 3, Randomized, Placebo-controlled, Double-blind, Adaptive Study to Evaluate the Safety and Efficacy of Inhaled Treprostinil in Patients with Pulmonary Hypertension due to Chronic Obstructive Pulmonary Disease (PH-COPD)

Sponsor: Lung Biotechnology
Principal Investigator: Barbara LeVarge, MD

This is a multicenter, randomized, double-blind, placebo-controlled, 30-week, adaptive cross-over study, with a Treatment Period of approximately 26 weeks under the Original Design or, if applicable, a 17-week parallel study, with a Treatment Period of approximately 14 weeks under the Contingent Design

Contact: Samantha Earnhardt, MPH CCRC  & Paula Glover, BA

TRACE: A multi-center, double-blind, placebo-controlled, Phase 4 study in patients with pulmonary arterial hypertension to assess the effect of selexipag on daily life physical activity and patient’s self-reported symptoms and their impacts

Sponsor: Actelion Pharmaceuticals
Principal Investigator: Barbara LeVarge, MD

The primary objective of this study is to evaluate the effect of selexipag on the physical activity of patients with pulmonary arterial hypertension (PAH) in their daily life, by using a wearable wrist device (actigraph). The actigraph will collect data on daily life physical activity in the patient’s real environment. In addition, the PAH symptoms and their impacts will be assessed by using an electronic patient reported outcome measure in the patient’s real environment. Patients will be assigned randomly to either selexipag or placebo.

Contact: Samantha Earnhardt, MPH CCRC  & Paula Glover, BA

PULSE-PHPF-001 entitled “A Phase 2B, Randomized, Double-Blind, Placebo-Controlled Dose Escalation Clinical Study to Assess the Safety and Efficacy of Pulsed, Inhaled Nitric Oxide (iNO) in Subjects with Pulmonary Hypertension Associated with Pulmonary Fibrosis on Long Term Oxygen Therapy (Part 1 and Part 2)

Sponsor: Bellerophon Therapeutics
Principal Investigator: Sheila Krishnan, DO

The primary objective in this study is to evaluate the efficacy and optimal dose of iNO on exercise using 6-minute walk distance (6MWD) in subjects with PH-PF currently receiving treatment with LTOT

Contact: Samantha Earnhardt, MPH CCRC  & Paula Glover, BA

Sarcoidosis

No current studies at this time.

Interstitial Lung Disease

Idiopathic Pulmonary Fibrosis Prospective Outcomes (IPF-PRO) and Chronic Fibrosing Interstitial Lung Disease With Progressive Phenotype Prospective Outcomes (IPF-PRO/ILD-PRO) Registry

Sponsor: Boehringer Ingelheim
Principal Investigator: Jason Lobo, MD

This registry will collect data on the strategies used to achieve a diagnosis of Idiopathic Pulmonary Fibrosis (IPF) and Chronic Fibrosing Interstitial Lung Disease with Progressive Phenotype (ILD) and the treatment and management efforts applied throughout study follow-up, clinical outcome events and patient reported outcome data. Blood samples will be collected periodically throughout the study for use in future research efforts. For participants with non-IPF, chronic fibrosing ILD with progressive phenotype, HRCT images will be collected throughout the study for use in future research efforts.

Contact:  Sonya Capps

Study of Pulmonary Rehabilitation In Nintedanib Treated Patients With IPF: Improvements in Activity, Exercise Endurance Time, and QoL

Sponsor: Boehringer Ingelheim
Principal Investigator: Jason Lobo, MD

The main objectives of this study are:

  • Determine the difference in change from baseline in Six Minute Walk Distance (6MWD) when pulmonary rehabilitation (PR) is added to stable underlying nintedanib therapy in patients with idiopathic pulmonary fibrosis (IPF)
  • Determine the difference in change in Quality of Life (QoL) when pulmonary rehabilitation (PR) is added to stable underlying nintedanib therapy in patients with idiopathic pulmonary fibrosis (IPF)
  • Determine if there is an enduring effect in 6MWD, QoL and lung function from pulmonary rehabilitation (PR) when pulmonary rehabilitation (PR) is added to stable underlying nintedanib therapy in patients with idiopathic pulmonary fibrosis (IPF)

Contact: Samantha Earnhardt, MPH CCRC  & Sonya Capps